Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Kastle Therapeutics

Status and phase: Completed; Phase 2

Conditions: Hypercholesterolemia, Familial

Treatments: Drug: ISIS 301012

Study type: Interventional

Funder types: Industry

Identifiers

NCT00280995

301012CS8

EudraCT No.: 2005-004796-38

Details and patient eligibility

About

The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 as add-on therapy in subjects with Homozygous Familial Hypercholesterolemia

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Weight ≥ 40 kg for Cohorts A, B, & C; Weight ≥ 50 kg for Cohort D
• Diagnosis of Homozygous Familial Hypercholesterolemia.
• Female must be non-pregnant and non-lactating.
• On stable lipid lowering therapy for at least 4 weeks.
• Lipid values that meet the pre-specified criteria.

Exclusion criteria

• Subject had heart problems in the prior 6 months.
• Subject has elevated ALT, AST, or CPK.
• History of renal disease, liver disease, or malignancy.
• Use of oral anticoagulants, unless the dose has been stable for 4 weeks
• Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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12 participants in 4 patient groups

Cohort A

Experimental group

Description:

Loading doses followed by weekly maintenance doses

Treatment:

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Cohort B

Experimental group

Description:

Loading doses followed by weekly maintenance doses

Treatment:

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Cohort C

Experimental group

Description:

Loading doses followed by weekly maintenance doses

Treatment:

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Cohort D

Experimental group

Description:

Loading doses followed by extended weekly maintenance doses

Treatment:

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012

Drug: ISIS 301012